Spark adds fresh evidence that pioneering gene therapy could be a game changer for patients
Spark Therapeutics has added another impressive round of late-stage data demonstrating that its gene therapy for an inherited eye disease could be a game changer for patients. This latest readout included positive outcomes for 9 crossover patients who received voretigene neparvovec (SPK-RPE65) a year ago, along with promising longer term results on the original group of 20 patients.
Nine patients with inherited retinal disease caused by mutations in the RPE65 gene were put in the control arm of the study, and all volunteered for the gene therapy after investigators concluded that the original treatment group had benefited markedly with significant gains in vision. A year after treatment, Spark says that 8 of the 9 “demonstrated the maximum improvement measurable on the primary endpoint at one year.” And the first 20 patients who were treated with their gene therapy have demonstrated a sustained benefit in vision, speaking to the durability of the change.
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